With the in­ter­im analy­sis for its Phase II Duchenne mus­cu­lar dy­s­tro­phy, lit­tle Capri­cor Ther­a­peu­tics was mere­ly look­ing for a guide­post, a sig­nal, a trend that the ef­fects seen in an ear­li­er tri­al can be repli­cat­ed with a new dos­ing reg­i­men and de­liv­ery method. In­stead, the biotech found it­self blessed with a bas­ket of da­ta points that seem to buck the nat­ur­al his­to­ry trend and sep­a­rate the drug from place­bo in a sta­tis­ti­cal­ly sig­nif­i­cant way, more than dou­bling its tiny stock $CAPR in pre-mar­ket trad­ing.

Lin­da Mar­bán Capri­cor

The pri­ma­ry end­point for the HOPE-2 tri­al is PUL 2.0, a mea­sure of up­per limb func­tion and one of sev­er­al ways Capri­cor quan­ti­fied skele­tal mus­cle im­prove­ment. Ex­ecs al­so spot­light­ed pul­monary and car­diac ben­e­fits in the top-line re­sults — fac­tors that are par­tic­u­lar­ly for the pa­tient pop­u­la­tion that they are tar­get­ing, who tend to be old­er and non-am­bu­la­to­ry.

Con­sist­ing of prog­en­i­tor cells de­rived from donor hearts, CAP-1002 is de­signed to re­lease ex­o­somes that kick off a cy­cle of mus­cle re­pair by sup­press­ing in­flam­ma­tion and dri­ving im­munomod­u­la­tion.

Cur­rent­ly, a num­ber of DMD pa­tients take glu­co­cor­ti­cos­teroids for that pur­pose, in­clud­ing pred­nisone and de­flaza­cort (sold in the US by Marathon as Em­flaza). There’s al­so Sarep­ta’s ex­on-skip­ping drug, eteplirsen (Ex­ondys 51), though its ben­e­fits have yet to be borne out; Sarep­ta and a hand­ful of ri­vals are now in hot pur­suit of a sup­posed one-time cure to re­place the miss­ing dy­s­trophin gene in pa­tients.

“While there had been ad­vances in gene ther­a­py, we be­lieve that con­trol­ling in­flam­ma­tion over and above what can be done by steroids is im­por­tant,” CEO Lin­da Mar­bán said in a con­fer­ence call.

Her hus­band, Ed­uar­do Mar­bán, had de­vel­oped the tech at Johns Hop­kins.

Da­ta from the small, place­bo-con­trolled study sug­gest that with a high­er dose than the past and de­liv­ered in­tra­venous­ly, the ther­a­py is work­ing. At the six-month cut, 6 pa­tients were evalu­able in each arm. For those treat­ed with CAP1002 mid-lev­el PUL — think mov­ing el­bows and pulling hand to mouth — de­te­ri­o­rat­ed by an av­er­age of 0.2 on a 6-point scale, com­pared to 0.8 on place­bo. The p-val­ue was 0.0389.

This and grip strength are the on­ly im­prove­ments still sta­tis­ti­cal­ly sig­nif­i­cant af­ter six months (there was al­so a three-month snap­shot), but Capri­cor stressed pos­i­tive trends through­out.

“I’d like to just re­mind peo­ple that this pop­u­la­tion of DMD re­al­ly rep­re­sents more than half of all DMD pa­tients and there re­al­ly have not been ther­a­pies that have re­al­ly fo­cused on this pop­u­la­tion which have just in­cred­i­bly high bur­den of dis­ease,” Craig Mc­Don­ald, the na­tion­al PI and a pro­fes­sor at UC Davis, said in the call.

On the key is­sue of safe­ty, Capri­cor not­ed that there had on­ly been one se­ri­ous ad­verse event among 30 in­fu­sions af­ter it put a stan­dard pre-med­ica­tion reg­i­men in place to mit­i­gate po­ten­tial im­mune re­ac­tions. Two pa­tients had had im­me­di­ate im­mune re­ac­tions to the treat­ment late last year, which prompt­ed the com­pa­ny to put a vol­un­tary hold on the tri­al.

Armed with these num­bers and an RMAT des­ig­na­tion from the FDA, Capri­cor is now look­ing in­to a “lean, ef­fi­cient” piv­otal study to po­si­tion the cell ther­a­py for a BLA — if it can find the cash to do so.

A few months ago it was forced to chop 21 staffers, or half of its work­force, in or­der to car­ry on with clin­i­cal ac­tiv­i­ties un­til the end of this year.

So­cial im­age: Shut­ter­stock

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll